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BOC Sciences |
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+1-631-485-4226 |
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SRP-4053 manufacturers
- Golodirsen
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2026-07-22
- CAS:1422959-91-8
- Min. Order: 1kg
- Purity: 98
- Supply Ability: 1000kgs
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| | SRP-4053 Basic information |
| Product Name: | SRP-4053 | | Synonyms: | SRP-4053;Golodirsen;RNA, [P-deoxy-P-(dimethylamino)](2′,3′-dideoxy-2′,3′-imino-2′,3′-seco)(2′a→5′)(G-m5U-m5U-G-C-C-m5U-C-C-G-G-m5U-m5U-C-m5U-G-A-A-G-G-m5U-G-m5U-m5U-C), 5′-[P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylphosphonamidate] (ACI) | | CAS: | 1422959-91-8 | | MF: | | | MW: | 0 | | EINECS: | | | Product Categories: | | | Mol File: | Mol File | ![SRP-4053 Structure]() |
| | SRP-4053 Chemical Properties |
| form | Solid | | color | White to off-white | | Sequence | RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(G-m5U-m5U-G-C-C-m5U-C-C-G-G-m5U-m5U-C-m5U-G-A-A-GG-m5U-G-m5U-m5U-C), 5'-[P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,Ndimethylphosphonamidate] |
| | SRP-4053 Usage And Synthesis |
| Uses | Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) that specifically targets exon 53 of dystrophin pre-mRNA. Golodirsen can be used for the research of Duchenne muscular dystrophy (DMD)[1][2]. | | References | [1] Heo YA. Golodirsen: First Approval. Drugs. 2020 Feb;80(3):329-333. DOI:10.1007/s40265-020-01267-2
[2] Scaglioni D, et, al. The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy. Acta Neuropathol Commun. 2021 Jan 6;9(1):7. DOI:10.1186/s40478-020-01106-1 |
| | SRP-4053 Preparation Products And Raw materials |
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